A clinical, molecular genetics and pathological study of a FTDP-17 family with a heterozygous splicing variant c.823-10G>T at the intron 9/exon 10 of the MAPT gene.


Journal

Neurobiology of aging
ISSN: 1558-1497
Titre abrégé: Neurobiol Aging
Pays: United States
ID NLM: 8100437

Informations de publication

Date de publication:
10 2021
Historique:
received: 23 12 2020
revised: 17 04 2021
accepted: 13 05 2021
pubmed: 19 7 2021
medline: 1 1 2022
entrez: 18 7 2021
Statut: ppublish

Résumé

We report the first clinical-radiological-genetic-molecular-pathological study of a kindred with c.823-10G>T MAPT intronic variant (rs63749974) associated with frontotemporal dementia and parkinsonism linked to chromosome 17 (FTDP-17). We describe the clinical spectrum within this family and emphasize the association between MAPT gene variants and motor neuron disease. This report of a second family with FTDP-17 associated with c.823-10G>T MAPT variant strongly supports pathogenicity of the variant and confirms it is a 4-repeat (4R) tauopathy. This intronic point mutation, probably strengthens the polypyrimidine tract and alters the splicing of exon 10 (10 nucleotides into intron 9) close to the 3' splice site.

Identifiants

pubmed: 34274155
pii: S0197-4580(21)00171-8
doi: 10.1016/j.neurobiolaging.2021.05.010
pii:
doi:

Substances chimiques

MAPT protein, human 0
tau Proteins 0

Types de publication

Journal Article Research Support, N.I.H., Extramural Research Support, Non-U.S. Gov't

Langues

eng

Sous-ensembles de citation

IM

Pagination

343.e1-343.e8

Subventions

Organisme : NINDS NIH HHS
ID : R01 NS076837
Pays : United States

Informations de copyright

Copyright © 2021. Published by Elsevier Inc.

Auteurs

Diana A Olszewska (DA)

Department of Neurology, Dublin Neurological Institute, Mater Misericordiae University Hospital, Dublin, Ireland.

Conor Fearon (C)

Department of Neurology, Dublin Neurological Institute, Mater Misericordiae University Hospital, Dublin, Ireland.

Christopher McGuigan (C)

Department of Neurology, St Vincent's University Hospital, Dublin, Ireland.

Terri P McVeigh (TP)

Our Lady's Children's Hospital, Crumlin, Dublin, Ireland.

Henry Houlden (H)

Department of Neuromuscular Diseases, UCL Queen Square Institute of Neurology, London, UK.

James M Polke (JM)

Department of Neuromuscular Diseases, UCL Queen Square Institute of Neurology, London, UK.

Brian Lawlor (B)

Department of Neuromuscular Diseases, UCL Queen Square Institute of Neurology, London, UK.

Robert Coen (R)

Mercer's Institute of Aging, St James's Hospital Dublin, Ireland.

Michael Hutchinson (M)

Mercer's Institute of Aging, St James's Hospital Dublin, Ireland.

Michael Hutton (M)

Department of Neurology, St Vincent's University Hospital, Dublin, Ireland.

Alan Beausang (A)

Eli Lilly Research Laboratories, Lilly Corporate Center, Indianapolis, USA.

Isabelle Delon (I)

Department of Neuropathology, Beaumont Hospital, Dublin, Ireland.

Francesca Brett (F)

East Genomic Laboratory Hub, Cambridge University Hospital NHS Foundation Trust, Addenbrooke's Treatment Centre, Hills Road, Cambridge, UK.

Ioanna Sevastou (I)

Department of Neuropathology, Beaumont Hospital, Dublin, Ireland.

Nuria Seto-Salvia (N)

East Genomic Laboratory Hub, Cambridge University Hospital NHS Foundation Trust, Addenbrooke's Treatment Centre, Hills Road, Cambridge, UK.

Rohan de Silva (R)

Department of Clinical and Movement Neuroscience, Reta Lila Weston Institute, UCL Queen Square Institute of Neurology, London, UK.

Tim Lynch (T)

Department of Neurology, Dublin Neurological Institute, Mater Misericordiae University Hospital, Dublin, Ireland; Health affairs, University College Dublin, Dublin, Ireland; Ireland East Hospital Group, Dublin, Ireland. Electronic address: tlynch@dni.ie.

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Classifications MeSH